BR112018015164A2 - single stranded oligonucleotide - Google Patents
single stranded oligonucleotideInfo
- Publication number
- BR112018015164A2 BR112018015164A2 BR112018015164A BR112018015164A BR112018015164A2 BR 112018015164 A2 BR112018015164 A2 BR 112018015164A2 BR 112018015164 A BR112018015164 A BR 112018015164A BR 112018015164 A BR112018015164 A BR 112018015164A BR 112018015164 A2 BR112018015164 A2 BR 112018015164A2
- Authority
- BR
- Brazil
- Prior art keywords
- nucleotide sequence
- oligonucleotide
- single stranded
- hybridization
- stranded oligonucleotide
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 6
- 125000003729 nucleotide group Chemical group 0.000 abstract 10
- 239000002773 nucleotide Substances 0.000 abstract 9
- 238000009396 hybridization Methods 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 1
- 108091028664 Ribonucleotide Proteins 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 230000004962 physiological condition Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 239000002336 ribonucleotide Substances 0.000 abstract 1
- 125000002652 ribonucleotide group Chemical group 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12P19/00—Preparation of compounds containing saccharide radicals
- C12P19/26—Preparation of nitrogen-containing carbohydrates
- C12P19/28—N-glycosides
- C12P19/30—Nucleotides
- C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03016—Phosphoprotein phosphatase (3.1.3.16), i.e. calcineurin
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03048—Protein-tyrosine-phosphatase (3.1.3.48)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03067—Phosphatidylinositol-3,4,5-trisphosphate 3-phosphatase (3.1.3.67)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- Health & Medical Sciences (AREA)
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- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
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- Animal Behavior & Ethology (AREA)
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- Plant Pathology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Obesity (AREA)
- Diabetes (AREA)
- Communicable Diseases (AREA)
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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Abstract
a presente invenção refere-se a um oligonucleotídeo de fita simples que é capaz de controlar um gene alvo com alta eficiência e pode ser facilmente produzido. o oligonucleotídeo de fita simples é representado pela fórmula x-l-y onde x e y hibridizam através de uma primeira porção de sequência de nucleotídeos e uma segunda porção de sequência de nucleotídeos. x é composto por 7 a 100 nucleotídeos, contém pelo menos um nucleotídeo modificado, e tem uma primeira sequência de nucleotídeos que é capaz de hibridização com um segundo oligonucleotídeo e contém pelo menos quatro nucleotídeos contíguos reconhecidos por rnase h. y é composto por 4 a 100 nucleotídeos, e tem uma segunda sequência de nucleotídeos que é capaz de hibridização com um segundo oligonucleotídeo e contém pelo menos um ribonucleotídeo. pelo menos uma de sequência de nucleotídeos x e sequência de nucleotídeos y tem uma sequência antissentido capaz de hibridização com um rna alvo. l é um grupo derivado de um terceiro oligonucleotídeo que é degradado sob condições fisiológicas.The present invention relates to a single stranded oligonucleotide that is capable of controlling a target gene with high efficiency and can be readily produced. The single stranded oligonucleotide is represented by the formula x-1-y where x and y hybridize through a first nucleotide sequence portion and a second nucleotide sequence portion. x is composed of 7 to 100 nucleotides, contains at least one modified nucleotide, and has a first nucleotide sequence that is capable of hybridization with a second oligonucleotide and contains at least four contiguous nucleotides recognized by rase h. y is comprised of 4 to 100 nucleotides, and has a second nucleotide sequence that is capable of hybridization to a second oligonucleotide and contains at least one ribonucleotide. at least one of nucleotide sequence x and nucleotide sequence y has an antisense sequence capable of hybridization to a target rna. 1 is a group derived from a third oligonucleotide that is degraded under physiological conditions.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2016012804 | 2016-01-26 | ||
JP2016158833 | 2016-08-12 | ||
PCT/JP2017/002831 WO2017131124A1 (en) | 2016-01-26 | 2017-01-26 | Single-stranded oligonucleotide |
Publications (1)
Publication Number | Publication Date |
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BR112018015164A2 true BR112018015164A2 (en) | 2018-12-26 |
Family
ID=59398721
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112018015164A BR112018015164A2 (en) | 2016-01-26 | 2017-01-26 | single stranded oligonucleotide |
Country Status (10)
Country | Link |
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US (2) | US11530409B2 (en) |
EP (1) | EP3409779A4 (en) |
JP (3) | JP6988481B2 (en) |
CN (2) | CN108884462B (en) |
AU (1) | AU2017211461B2 (en) |
BR (1) | BR112018015164A2 (en) |
CA (1) | CA3023222A1 (en) |
MX (2) | MX2018009090A (en) |
TW (2) | TWI803455B (en) |
WO (1) | WO2017131124A1 (en) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2017131124A1 (en) * | 2016-01-26 | 2017-08-03 | 日産化学工業株式会社 | Single-stranded oligonucleotide |
TWI830693B (en) | 2017-02-06 | 2024-02-01 | 日商日產化學工業股份有限公司 | single stranded oligonucleotide |
CA3070809A1 (en) | 2017-07-26 | 2019-01-31 | Nissan Chemical Corporation | Single-stranded oligonucleotide |
AU2019237599A1 (en) | 2018-03-20 | 2020-11-12 | Nissan Chemical Corporation | Antisense oligonucleotide having reduced toxicity |
EP3981431A4 (en) | 2019-03-14 | 2023-06-28 | Rena Therapeutics Inc. | Nucleic acid complex for modulating ihh expression |
EP4098747A1 (en) | 2020-01-31 | 2022-12-07 | Sanwa Kagaku Kenkyusho Co., Ltd. | Antisense oligonucleotide of atn1 |
WO2021177418A1 (en) | 2020-03-04 | 2021-09-10 | 日産化学株式会社 | Antisense oligonucleotide of calm2 |
JPWO2022211129A1 (en) * | 2021-04-01 | 2022-10-06 | ||
EP4353266A1 (en) | 2021-05-31 | 2024-04-17 | Rena Therapeutics Inc. | Ligand-bound nucleic acid complex |
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CN116064544A (en) | 2023-05-05 |
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EP3409779A1 (en) | 2018-12-05 |
TWI803455B (en) | 2023-06-01 |
JP7435583B2 (en) | 2024-02-21 |
WO2017131124A1 (en) | 2017-08-03 |
JP6988481B2 (en) | 2022-01-05 |
TW201738376A (en) | 2017-11-01 |
US11530409B2 (en) | 2022-12-20 |
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US20230203498A1 (en) | 2023-06-29 |
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